StemEx®, Receives Orphan Medicinal Product Designation In The EU - Treatment Of Acute Lymphoblastic Leukemia And Acute Myeloid Leukemia

Main Category: Lymphoma / Leukemia / Myeloma
Also Included In: Blood / Hematology;  Regulatory Affairs / Drug Approvals;  Cancer / Oncology
Article Date: 12 Mar 2009 - 6:00 PDT

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The Gamida Cell-Teva Joint Venture announced today that orphan designation was granted by the European Commission for the investigational medicinal product StemEx® for the treatment of acute lymphoblastic leukemia and acute myeloid leukemia. The European Commission consequently follows the positive opinion of the European Medicine Agency's Orphan Medicinal Products Committee. The FDA granted StemEx orphan drug designation in March 2005 for use as hematopoietic support in patients with relapsed or refractory hematologic malignancies (cancer of the blood system) who are receiving high dose therapy.

"This is very important news. Having an orphan drug designation for StemEx in the U.S. and now in Europe affords an impressive array of financial and product development benefits. Today's news moves StemEx one step closer to market launch," said Dr. Yael Margolin, CEO of Gamida Cell.

By EU regulation, orphan medicinal products are intended for the diagnosis, prevention or treatment of life-threatening or chronically debilitating conditions that affect no more than five in 10,000 people in the European Union, or are medicines which, for economic reasons, would be unlikely to be developed without incentives. Benefits include, among others, market exclusivity in the European Union for 10 years from the date of the orphan medicinal product designation and an EMEA fee-reduction for the drug.

StemEx is a graft of expanded stem/progenitor cells, derived from a single unit of umbilical cord blood and transplanted in combination with non expanded cells from the same unit. It is currently being researched as an alternative to a bone marrow transplant, for patients with certain blood cancers, in an advanced Phase III, pivotal study called ExCell now in progress at prestigious cord blood transplantation clinical sites in the U.S., Europe and Israel. For more information about the ExCell trial as well as participating sites, please visit http://www.stemexstudy.com.

"The European orphan medicinal product designation by the European Commission further validates the potential of StemEx as a therapeutic treatment for blood cancers. We look forward to continuing our work in cooperation with the EMEA with the goal of providing an alternative treatment to bone marrow transplantation for adolescents and adults," said Michal Austin, director of regulatory affairs.

About Gamida Cell

Gamida Cell Ltd. is a world leader in stem cell expansion technologies and therapeutic products. The company is developing a pipeline of products in stem cell transplantation and in tissue regeneration to effectively treat debilitating and often fatal illnesses such as cancer, hematological diseases, autoimmune diseases and ischemic diseases. Gamida Cell's therapeutic candidates contain populations of adult stem cells, selected from non-controversial sources such as umbilical cord blood and bone marrow, which are expanded in culture. Gamida Cell was successful in translating these proprietary expansion technologies into robust and validated manufacturing processes under GMP. Gamida Cell's first product, StemEx for blood cancers, is anticipated to reach the market in 2011. Gamida Cell's current shareholders include: Elbit Imaging, Biomedical Investment, Israel Healthcare Venture, Teva Pharmaceuticals, Amgen, Denali Ventures and Auriga Ventures. For more information, please visit: http://www.gamida-cell.com.

Gamida Cell